JP2020522265A - オリゴヌクレオチド組成物及びその使用方法 - Google Patents

オリゴヌクレオチド組成物及びその使用方法 Download PDF

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JP2020522265A
JP2020522265A JP2019566837A JP2019566837A JP2020522265A JP 2020522265 A JP2020522265 A JP 2020522265A JP 2019566837 A JP2019566837 A JP 2019566837A JP 2019566837 A JP2019566837 A JP 2019566837A JP 2020522265 A JP2020522265 A JP 2020522265A
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oligonucleotide
independently
alkyl
oligonucleotides
pattern
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JP2020522265A5 (https=
Inventor
ヴァージェシー，チャンドラ
イワモト，ナオキ
チャールズ ドネル バトラー，デイビット
チャールズ ドネル バトラー，デイビット
マラッパン，スブラマニアン
ル，ゲンリャン
ジンシン チャン，ジェイソン
ジンシン チャン，ジェイソン
ヴァシパディーカル，ヴィノッド
デイビッド フランク−カメネットスキー，マリア
デイビッド フランク−カメネットスキー，マリア
エンリケ アッポニ，ルチアーノ
エンリケ アッポニ，ルチアーノ
マリア ウィスニウスカ，ハンナ
マリア ウィスニウスカ，ハンナ
チェン，シアユン
ジン チョ，ヤング
ジン チョ，ヤング
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Wave Life Sciences Pte Ltd
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Wave Life Sciences Pte Ltd
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Publication of JP2020522265A publication Critical patent/JP2020522265A/ja
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Priority to JP2023125286A priority Critical patent/JP2023153920A/ja
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    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C—CHEMISTRY; METALLURGY
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
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    • C07H17/00—Compounds containing heterocyclic radicals directly attached to hetero atoms of saccharide radicals
    • C07H17/04—Heterocyclic radicals containing only oxygen as ring hetero atoms
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
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    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/30—Chemical structure
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    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/30—Chemical structure
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    • C12N2310/3519—Fusion with another nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers

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JP2019566837A 2017-06-02 2018-06-01 オリゴヌクレオチド組成物及びその使用方法 Pending JP2020522265A (ja)

Priority Applications (1)

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JP2023125286A JP2023153920A (ja) 2017-06-02 2023-08-01 オリゴヌクレオチド組成物及びその使用方法

Applications Claiming Priority (5)

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US201762514769P 2017-06-02 2017-06-02
US62/514,769 2017-06-02
US201862670702P 2018-05-11 2018-05-11
US62/670,702 2018-05-11
PCT/US2018/035712 WO2018223073A1 (en) 2017-06-02 2018-06-01 Oligonucleotide compositions and methods of use thereof

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JP2020522265A5 JP2020522265A5 (https=) 2021-08-19

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US (3) US20210198305A1 (https=)
EP (1) EP3630788A4 (https=)
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CN (2) CN120330183A (https=)
TW (1) TW201908483A (https=)
WO (1) WO2018223073A1 (https=)

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